The trial, led by Professor Jun Takahashi at Kyoto University in Japan, follows on from the successful application of this treatment in a primate model of the disease reported last year (see BioNews 916). Seven patients will be treated with the 'reprogrammed' adult stem cells and followed up for two years, with the primary objective to assess safety and tolerability.
'This will be the world’s first clinical trial using iPS cells on Parkinson's disease,' Professor Takahashi told a news conference last week.
The treatment itself will consist of an injection of 5 million stem cell-derived neural progenitor cells, directly into the brain of patients. The researchers hope that over time, the progenitor cells will mature and engraft into the patients' neural networks and begin to release dopamine, which is the neurotransmitter lost in Parkinson's disease.
Typically, the loss of dopaminergic neurons in Parkinson's disease causes patients to have difficulty initiating movement and sees them develop a consistent baseline tremor, both of which are very debilitating.
The stem cells used in these treatments will come from healthy third-party donor blood cells which are prepared and banked at the iPS Cell Stock for Regenerative Medicine. Although this carries a risk of transplant rejection, the researchers state that 'using stocks of cells, we can proceed much more quickly and cost-effectively'. The patients will also be treated with a common immunosuppressant to alleviate this risk.
The trial will be recruiting Japanese residents. If successful, the research team hopes it will become a treatment covered by national health insurance.
Kosei Hasegawa, representative chairman of the Japan Parkinson's Disease Association, told The Japan Times: 'I want the method to be established as a treatment available for anyone as soon as possible.'